uniQure Submits BLA for Huntington's Gene Therapy, Seeks Priority Review
QURE has more than doubled off its 52-week low of $8.73 on light trading volume (0.4× avg).
Summary
uniQure has submitted its Biologics License Application to the FDA for ifezuntirgene inilparvovec (AMT-130) in Huntington's disease, requesting priority review. The company also submitted a Marketing Authorisation Application to the UK MHRA. This follows the FDA's June agreement to accept 3-year Phase I/II data as the primary basis for a BLA, and the Q2 2026 announcement of alignment on an accelerated approval pathway. The submission is a major regulatory milestone for a disease with no approved therapies to slow progression. If priority review is granted, the FDA review cycle could be shortened to six months after the 60-day filing review period. The company plans to present four-year Phase I/II data before the end of Q3 2026.
At the time of this announcement, QURE was trading at $49.00 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $3.3B. The 52-week trading range was $8.73 to $71.50. This news item was assessed with positive market sentiment and an importance score of 8 out of 10. Source: GlobeNewswire.